Glanzmann's Thrombasthenia Treatment: A Prospective Observational Registry on the Use of Recombinant Human Activated Factor VII and Other Hemostatic Agents

Glanzmann's thrombasthenia (GT) is a rare congenital bleeding disorder caused by deficiency or dysfunction of platelet surface glycoprotein (GP) IIb/IIIa receptor. Platelet transfusion is the standard treatment for bleeding that remains non-responsive to conservative measures, and for surgical coverage. Platelet transfusions, however, may result in the development of antibodies to GPIIb/IIIa and/or human leukocyte antigen (HLA), rendering further transfusions ineffective. Recombinant human activated factor VII (rFVIIa; NovoSeven®/Niastase® [Canada], Novo Nordisk, Bagsvaerd, Denmark) has documented efficacy in GT patients, and is approved in the European Union for the treatment of GT patients with platelet antibodies and platelet refractoriness. However, there are insufficient data to determine the optimal rFVIIa regimen (eg, for major surgery) or to allow thorough safety evaluation (eg, thrombotic risk). A post-marketing, prospective, observational, multinational registry has been developed to collect data on the efficacy and safety of rFVIIa in the treatment and prevention of bleeding in GT patients with platelet antibodies or platelet refractoriness. Patients treated with other hemostatic agents or rFVIIa to avoid the development of antibodies against GPIIb/IIIa will also be reported. Standardized data will be collected using a customized internet-based (www.glanzmann-reg.org) data collection tool. Data collection will begin in 2005 and continue for up to 6 years. Patients of all ages from any country are eligible for inclusion.